A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
Study Details
Study Description
Brief Summary
This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Understanding the natural history of the disease is not only essential to monitoring and comparing patient populations in clinical trials. It is also fundamental in the predevelopment phase in order to optimize the study duration needed to observe a statistically significant outcome. Furthermore, since the progression of RP is usually slow, relying on traditional tests can take an unfeasible length of time to observe any meaningful changes and assess therapeutic efficacy for new drugs. Therefore, the results of this study will be beneficial in establishing reliable endpoints and outcome measures for future clinical trials. Such outcome measures may be able to detect treatment response with more precision. More importantly, investigators may be able to detect changes early enough to prevent irreversible vision loss.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Retinitis Pigmentosa Patients with Retinitis Pigmentosa |
Outcome Measures
Primary Outcome Measures
- Change in mean macular sensitivity (dB) over time as assessed by microperimetry [Baseline, every six months up to 2 years]
Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula
Secondary Outcome Measures
- Change in Best Corrected Visual Acuity (BVCA) [Baseline, every six months up to 2 years]
Scoring will be determined by the number of letters gained or lost per month using Early Treatment Diabetic Retinopathy Study (ETDRS) Letter Score and visual acuity score together with an overall score range of 0 to 20/20 where 0 is the worst vision and 20/20 is the best.
- Change in Ellipsoid Zone (EZ) width [Baseline, every six months up to 2 years]
This will be assessed by spectral domain optical coherence tomography (SD-OCT)
- Change in Quality of Life survey metrics [Baseline, every year up to 2 years]
Scoring will be determined by the National Eye Institute's Visual Function Questionnaire (NEI-VFQ-25). It has 25 question elements each with score ranging from 1(excellent) to 6(very poor), therefore a total minimum score of 25 and maximum score 150.
- Change in mean retinal sensitivity [Baseline and at 2 years]
Static Octopus Perimetry will be used to test whether there is a change in mean retinal sensitivity over time using its 30-2 program with III target
- Correlation between change in visual functional and anatomical measures [Baseline, every six months up to 2 years]
Change in visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) will be correlated to anatomical parameters such as Ellipsoid width (measurement on Optical Coherence Tomography)
- Correlation between change in visual functional measures and Quality of Life survey metrics [Baseline, every year up to 2 years]
Change in Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25) will be compared to visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry)
Other Outcome Measures
- Correlation between baseline functional and anatomical measures [Baseline, up to 2 years]
Visual function parameters at baseline such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) will be correlated to anatomical parameters at baseline such as Ellipsoid width (measurement on Optical Coherence Tomography)
- Correlation between baseline functional measures and Quality of Life survey metrics [Baseline, up to 2 years]
Visual function parameters at baseline such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) will be correlated to baseline Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25)
- Correlation between functional, anatomic and Quality of Life measures [Baseline, up to 2 years]
Visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry), anatomical parameters such as Ellipsoid width (measurement on Optical Coherence Tomography) Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25) will be correlated.
- Proportion of eyes with ≥ 5 loci that show ≥ 6 decibels (dB) decline in mean macular sensitivity from baseline [Baseline, every six months up to 2 years]
This will be measured using MAIA microperimetry
- Proportion of eyes with ≥ 5 loci that show ≥ 7 decibels (dB) decline in mean retinal sensitivity from baseline [Baseline and at 2 years]
This will be measured by static Octopus perimetry using 30-2 program with III target
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Age 18 years or older
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Patients diagnosed with Retinitis Pigmentosa
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Ability to provide informed consent
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Ability to authorize use and disclosure of protected health information
Exclusion Criteria:
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Concomitant ocular pathology that limits central macular function, including but not limited to age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion
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If EZ width ≤200µm
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Wilmer Eye Institute at Johns Hopkins University | Baltimore | Maryland | United States | 21287 |
Sponsors and Collaborators
- Johns Hopkins University
Investigators
- Principal Investigator: Peter A Campochiaro, M.D., Johns Hopkins University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- IRB00227603